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NaPro Gene Editing Technology Highlighted in Three Scientific Journals.


Business Editors/Health/Medical Writers

BOULDER, Colo.--(BUSINESS WIRE)--March 6, 2003

NaPro BioTherapeutics, Inc. (Nasdaq:NPRO NPRO Non Planar Ring Oscillator
NPRO Naval Plant Representative Office
NPRO Naval Petroleum Reserves Office
NPRO Non-Process Run-Out (punch card readers) 
) today announced that recent publications in three scientific journals have validated the utility of several of its proprietary genomic tools covering a wide range of molecular research and potential therapeutic applications.

The announcement followed the publication of a series of studies by Eric B. Kmiec, Ph.D. and colleagues at the Delaware Biotechnology Institute The Biotechnology Institute is an independent nonprofit organization founded to teach the public about the benefits of biotechnology. It was created in 1998 by the biotechnology industry and is located in Arlington, Virginia.  at the University of Delaware [3] The student body at the University of Delaware is largely an undergraduate population. Delaware students have a great deal of access to work and internship opportunities.  in recent issues of three peer-reviewed scientific publications: Genetics (2003, 163: 527-538), Nucleic Acids Research Nucleic Acids Research or NAR is a peer reviewed scientific journal published by Oxford University Press. NAR publishes research on Nucleic Acids, such as DNA and RNA, and related work. Some of its content is available under and open access license.  (2003, 31:3, 899-910) and Biomolecular Engineering (2003, 20: 7-20).

The technology described in these publications was developed at the University of Delaware and is licensed exclusively to NaPro BioTherapeutics.

"The technology provides the foundation for new categories of research tools and therapeutics, including methods that can 'edit' genes to help treat certain diseases," stated Dr. Kmiec. "Together these studies indicate that with NaPro's platforms, researchers can tackle several major bottlenecks in functional genomics Noun 1. functional genomics - the branch of genomics that determines the biological function of the genes and their products
genomics - the branch of genetics that studies organisms in terms of their genomes (their full DNA sequences)
 research, including effecting gene repair with greater precision than ever before, by stimulating the natural repair mechanism of cells at the molecular level."

Jeffrey White, president of NaPro's Genomics Division, commented, "These studies provide further validation for critical elements of our Gene Editing platform, and the technology underlying our therapeutic development program in Sickle Cell Disease sickle cell disease or sickle cell anemia, inherited disorder of the blood in which the oxygen-carrying hemoglobin pigment in erythrocytes (red blood cells) is abnormal. ."

Mr. White continued, "The technology also affords a broad new range of research tools, including the rapid and cost-effective creation of SNPs or haplotypes in genes of interest. The technologies presented in these three papers are available today from NaPro, making it possible for researchers to order custom-designed genes modified to the single base pair level."

NaPro Gene Editing Technology Not Sequence Dependent, Genetics

Study Reports

The Genetics paper reported the success by Eric Kmiec and Erin Brachman in using specially modified single-stranded oligonucleotides to direct base changes in a target gene in a yeast strain. Utilizing DNA repair DNA repair refers to a collection of processes by which a cell identifies and corrects damage to the DNA molecules that encode its genome. In human cells, both normal metabolic activities and environmental factors such as UV light can cause DNA damage, resulting in as many as 1  enzymes, the researchers effected a change - through targeted nucleotide exchange - of the target mutant gene mutant gene
n.
A gene that has lost, gained, or exchanged some of the material it received from its parent, resulting in a permanent transmissible change in its function.
 to a "wild type," or normal status.

This approach confirmed the ability of single-stranded oligonucleotides to direct nucleotide exchange of various point mutations point mutation
n.
A mutation that involves a single nucleotide and may consist of loss of a nucleotide, substitution of one nucleotide for another, or the insertion of an additional nucleotide.
 irrespective of irrespective of
prep.
Without consideration of; regardless of.

irrespective of
preposition despite 
 their sequence context. The paper also reported that a critical DNA repair gene could enhance targeted nucleotide exchange up to 22 times normal.

"The data published in Genetics strongly affirm the universal ability of NaPro's proprietary oligonucleotides to direct highly specific nucleotide exchange. We anticipate this study will form the basis for a protein-driven nucleotide exchange strategy that will work in higher organisms," said Dr. Kmiec.

Stable Structure Identified as First Step in Gene Repair,

According to according to
prep.
1. As stated or indicated by; on the authority of: according to historians.

2. In keeping with: according to instructions.

3.
 Nucleic Acids Research Study

In the Nucleic Acids Research study, Dr. Kmiec and Miya Drury described a functional template, known as the double-displacement ("double-D") structure, or loop. This discovery occurred as Dr. Kmiec and his colleagues were investigating the mechanism for endogenous endogenous /en·dog·e·nous/ (en-doj´e-nus) produced within or caused by factors within the organism.

en·dog·e·nous
adj.
1. Originating or produced within an organism, tissue, or cell.
 repair of genetic mutations directed by modified single-stranded DNA DNA: see nucleic acid.
DNA
 or deoxyribonucleic acid

One of two types of nucleic acid (the other is RNA); a complex organic compound found in all living cells and many viruses. It is the chemical substance of genes.
 oligonucleotides in yeast, plant and mammalian cells.

"In addition to this structure being a necessary for first step of DNA pairing and gene repair, it also has broad application - primarily because of its stability and specificity for optimizing the process of isolation, detection, and cleavage," commented Dr. Kmiec.

More Efficient, Specific Two-Site Gene Editing Enabled Recently

Reported in Biomolecular Engineering Study

The third study, published in Biomolecular Engineering, reported results from research that may provide scientists with a better method for achieving gene editing. In the article, Dr. Kmiec et al described results of studies evaluating multiple site nucleotide exchange using single-stranded proprietary oligonucleotides.

"The goal," Dr. Kmiec noted, "was to generate nucleotide exchange events at multiple sites along a specific genetic sequence in a laboratory model. The study further demonstrated that these new oligonucleotides were able to direct targeted nucleotide exchange at two independent sites, a reaction characteristic that may be useful for many genomics applications."

About NaPro BioTherapeutics

NaPro BioTherapeutics, Inc. is a pharmaceutical company focused in two distinct research, development and marketing areas: the development and in-licensing of novel pharmaceutical products, primarily in the area of anti-cancer agents, and the development of novel genomic technologies, primarily in the area of "gene editing," for application in human therapeutics, diagnostics, agribiotechnology, and pharmacogenomics Pharmacogenomics is the branch of pharmacology which deals with the influence of genetic variation on drug response in patients by correlating gene expression or single-nucleotide polymorphisms with a drug's efficacy or toxicity. .

The statements in this release that are not historical facts are forward-looking and represent management's beliefs and assumptions as of the date of this release based on currently available information. Forward-looking statements forward-looking statement

A projected financial statement based on management expectations. A forward-looking statement involves risks with regard to the accuracy of assumptions underlying the projections.
 can be identified by their predictions as to future events or by discussions of strategy.

Such statements involve risks and uncertainties including, but not limited to, the acceleration of our functional genomics service capabilities, our ability to provide timely and cost effective gene research tools and services, and our success in therapeutic applications of our Gene Editing technology.

Should these risks materialize, or should the underlying assumptions prove incorrect, actual results could differ materially from those included in the forward-looking statement.

Reference is made to other factors described in NaPro's documents filed from time to time with the Securities and Exchange Commission, including its Annual Report on Form 10-K Form 10-K

A report required by the SEC from exchange-listed companies that provides for annual disclosure of certain financial information.


Form 10-K

See 10-K.
 for the year ended December 31, 2001 and Amendment No. 4 to its registration statement filed with the Securities and Exchange Commission on July 1, 2002.

NaPro disclaims any intention or obligation to update publicly or revise such statements whether as a result of new information, future events or otherwise.
COPYRIGHT 2003 Business Wire
No portion of this article can be reproduced without the express written permission from the copyright holder.
Copyright 2003, Gale Group. All rights reserved. Gale Group is a Thomson Corporation Company.

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Geographic Code:1USA
Date:Mar 6, 2003
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